Healthy SubjectsPhase 1NCT02944487
A Study to Assess the Safety and Pharmacokinetics of Lucerastat (OGT 923) in Healthy Subjects
A Phase 1 study testing Lucerastat in people with Healthy Subjects, run by Idorsia Pharmaceuticals Ltd. at 1 site in United Kingdom.
Trial summary
The objectives of this study were to evaluate the safety and tolerability of lucerastat, and to determine its pharmacokinetic profile as single oral doses at different strengths. Read more about this study
- Who is it for?
- Ages 18 to 45 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Healthy Subjects
- What is being tested?
- Lucerastat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 39 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Oct 2002to Dec 2002
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- Men only
- Healthy volunteers accepted
- Adult
Who may be able to join
- Signed informed consent form.
- Male subjects aged from 18 to 45 years at screening.
- Body weight between 50 and 100 kg and body mass index (BMI) between 18.0 and 29.0 kg/m2 at screening.
- Healthy on the basis of physical examination, cardiovascular assessments and laboratory tests.
Who cannot join
- History or clinical evidence of any disease or medical / surgical condition or treatment, which may put the subject at risk of participation in the study or may interfere with the absorption, distribution, metabolism or excretion of the study treatments.
- Serious adverse reaction or hypersensitivity to any drug.
- Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomised, Double-blind, Placebo-controlled Ascending Dose Tolerance Study of OGT 923 in Healthy Male Volunteers
The objectives of this study were to evaluate the safety and tolerability of lucerastat, and to determine its pharmacokinetic profile as single oral doses at different strengths.
Read the detailed description
The subjects were enrolled sequentially to five dose groups, starting with the lowest dose level. Subjects could participate in only one Group.
- Conditions
- Healthy Subjects
- Being tested
- Lucerastat, Placebo
- Keywords
- safety, pharmacokinetics
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LucerastatDrug
Also called OGT-923, ACT-434964
Hard gelatin capsule for oral administration containing lucerastat
PlaceboDrug
Matching placebo capsules
| Study group | Role | What they receive |
|---|---|---|
| Single ascending doses of lucerastat | Experimental | Subjects were enrolled sequentially in 4 groups and received a single oral dose of lucerastat from 100 mg to 1000 mg in the morning of Day 1 Receives: Drug: Lucerastat |
| B.i.d. Dose Group | Experimental | Subjects received two doses of lucerastat (2 x 1 g) 12 hours apart on Day 1 Receives: Drug: Lucerastat |
| Placebo for singe ascending doses | Placebo comparator | These subjects received matching placebo administered orally in the morning of Day 1 Receives: Drug: Placebo |
| Placebo for b.i.d.Group | Placebo comparator | These subjects received matching placebo administered orally in the morning and in the evening of Day 1 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with Adverse Events (AEs)
An AE was defined as any untoward medical occurrence in a clinical investigation subject, which did not necessarily have a causal relationship with the treatment.
Measured: From baseline up to 7 days post-administration
Maximum plasma concentration (Cmax) of lucerastat after single ascending doses of lucerastat
Cmax was determined directly from the observed plasma concentration-time curves of lucerastat in subjects receiving a single dose.
Measured: PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration
Maximum plasma concentration (Cmax) of lucerastat after two daily doses of lucerastat
Cmax was determined directly from the observed plasma concentration-time curves of lucerastat in subjects receiving lucerastat twice daily.
Measured: PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration
Time to reach Cmax (tmax) of lucerastat after single ascending doses of lucerastat
tmax was determined directly from the observed plasma concentration-time curves of lucerastat in subjects receiving a single dose.
Measured: PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration
Time to reach Cmax (tmax) of lucerastat after two daily doses of lucerastat
tmax was determined directly from the observed plasma concentration-time curves of lucerastat in subjects receiving lucerastat twice daily.
Measured: PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration
Area under the plasma concentration-time curves (AUC) of lucerastat after single ascending doses of lucerastat
AUC was calculated from time zero to time t (last PK blood sample in which drug was detected) and extrapolated to infinity for subjects receiving a single dose.
Measured: PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration
Area under the plasma concentration-time curves (AUC) of lucerastat after two daily doses of lucerastat
AUC was calculated from time zero to time t (last PK blood sample in which drug was detected) and extrapolated to infinity for subjects receiving lucerastat twice daily
Measured: PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration
Terminal elimination half-life (t1/2) of lucerastat after single ascending doses of lucerastat
t1/2 was calculated from the corresponding plasma concentrations-time curves for subjects receiving a single dose
Measured: PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration
Terminal elimination half-life (t1/2) of lucerastat after two daily doses of lucerastat
t1/2 was calculated from the plasma concentrations-time curves for subjects receiving lucerastat twice daily
Measured: PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration
Secondary outcomes (4)
Change from baseline in heart rate
Measured: Up to 24 hours post administration
Change from baseline in blood pressure
Measured: Up to 24 hours post administration
Change from baseline in electrocardiogram (ECG) variables
Measured: Up to 24 hours post administration
Change from baseline in laboratory tests
Measured: Up to 24 hours post administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Oct 2016 (estimated)
- Study startOct 2002
- Primary completionDec 2002
- Study completionDec 2002
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Healthy SubjectsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Idorsia Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02944487
- Sponsor's protocol ID
- OGT923-001
- Status last verified
- Apr 2025
- First submitted
- 24 Oct 2016
- Last updated
- 1 May 2025
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Guerard N, Morand O, Dingemanse J. Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: safety, tolerability, and pharmacokinetics in healthy subjects. Orphanet J Rare Dis. 2017 Jan 14;12(1):9. doi: 10.1186/s13023-017-0565-9.
PubMed 28088251
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomised, Double-blind, Placebo-controlled Ascending Dose Tolerance Study of OGT 923 in Healthy Male Volunteers
- ClinicalTrials.gov ID
- NCT02944487
- Acronym / sponsor ID
- OGT923-001
- Conditions
- Healthy Subjects
- Keywords
- safety, pharmacokinetics
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.Industry (pharma, biotech, devices)
- Study start
- Oct 2002
- Primary completion
- Dec 2002 (actual)
- Study completion
- Dec 2002 (actual)
- First posted
- 26 Oct 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objectives of this study were to evaluate the safety and tolerability of lucerastat, and to determine its pharmacokinetic profile as single oral doses at different strengths.
Detailed description
The subjects were enrolled sequentially to five dose groups, starting with the lowest dose level. Subjects could participate in only one Group.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Single ascending doses of lucerastat | Experimental | Drug: LucerastatSubjects were enrolled sequentially in 4 groups and received a single oral dose of lucerastat from 100 mg to 1000 mg in the morning of Day 1 |
| B.i.d. Dose Group | Experimental | Drug: LucerastatSubjects received two doses of lucerastat (2 x 1 g) 12 hours apart on Day 1 |
| Placebo for singe ascending doses | Placebo comparator | Drug: PlaceboThese subjects received matching placebo administered orally in the morning of Day 1 |
| Placebo for b.i.d.Group | Placebo comparator | Drug: PlaceboThese subjects received matching placebo administered orally in the morning and in the evening of Day 1 |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Signed informed consent form.
- Male subjects aged from 18 to 45 years at screening.
- Body weight between 50 and 100 kg and body mass index (BMI) between 18.0 and 29.0 kg/m2 at screening.
- Healthy on the basis of physical examination, cardiovascular assessments and laboratory tests.
Key exclusion criteria (3 listed)
- History or clinical evidence of any disease or medical / surgical condition or treatment, which may put the subject at risk of participation in the study or may interfere with the absorption, distribution, metabolism or excretion of the study treatments.
- Serious adverse reaction or hypersensitivity to any drug.
- Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LucerastatAlso: OGT-923, ACT-434964Hard gelatin capsule for oral administration containing lucerastat | Drug | B.i.d. Dose Group, Single ascending doses of lucerastat |
| PlaceboMatching placebo capsules | Drug | Placebo for b.i.d.Group, Placebo for singe ascending doses |
Primary outcome
Number of participants with Adverse Events (AEs)
Time frame: From baseline up to 7 days post-administration
An AE was defined as any untoward medical occurrence in a clinical investigation subject, which did not necessarily have a causal relationship with the treatment.
Maximum plasma concentration (Cmax) of lucerastat after single ascending doses of lucerastat
Time frame: PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration
Cmax was determined directly from the observed plasma concentration-time curves of lucerastat in subjects receiving a single dose.
Maximum plasma concentration (Cmax) of lucerastat after two daily doses of lucerastat
Time frame: PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration
Cmax was determined directly from the observed plasma concentration-time curves of lucerastat in subjects receiving lucerastat twice daily.
Time to reach Cmax (tmax) of lucerastat after single ascending doses of lucerastat
Time frame: PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration
tmax was determined directly from the observed plasma concentration-time curves of lucerastat in subjects receiving a single dose.
Time to reach Cmax (tmax) of lucerastat after two daily doses of lucerastat
Time frame: PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration
tmax was determined directly from the observed plasma concentration-time curves of lucerastat in subjects receiving lucerastat twice daily.
Area under the plasma concentration-time curves (AUC) of lucerastat after single ascending doses of lucerastat
Time frame: PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration
AUC was calculated from time zero to time t (last PK blood sample in which drug was detected) and extrapolated to infinity for subjects receiving a single dose.
Area under the plasma concentration-time curves (AUC) of lucerastat after two daily doses of lucerastat
Time frame: PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration
AUC was calculated from time zero to time t (last PK blood sample in which drug was detected) and extrapolated to infinity for subjects receiving lucerastat twice daily
Terminal elimination half-life (t1/2) of lucerastat after single ascending doses of lucerastat
Time frame: PK Blood samples were collected at pre-dose and at scheduled time points up to 24 hours post administration
t1/2 was calculated from the corresponding plasma concentrations-time curves for subjects receiving a single dose
Terminal elimination half-life (t1/2) of lucerastat after two daily doses of lucerastat
Time frame: PK Blood samples were collected at pre-dose and at scheduled time points up to 48 hours after the first administration
t1/2 was calculated from the plasma concentrations-time curves for subjects receiving lucerastat twice daily
4 secondary outcomes
Change from baseline in heart rate
Time frame: Up to 24 hours post administration
Change from baseline in blood pressure
Time frame: Up to 24 hours post administration
Change from baseline in electrocardiogram (ECG) variables
Time frame: Up to 24 hours post administration
Change from baseline in laboratory tests
Time frame: Up to 24 hours post administration
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 2025.
- Derived from this study: PubMed 28088251Guerard N, Morand O, Dingemanse J. Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: safety, tolerability, and pharmacokinetics in healthy subjects. Orphanet J Rare Dis. 2017 Jan 14;12(1):9. doi: 10.1186/s13023-017-0565-9.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Idorsia Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Lucerastat in people with Healthy Subjects, run by Idorsia Pharmaceuticals Ltd. at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with Adverse Events (AEs) (time frame: From baseline up to 7 days post-administration). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02944487, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 May 2025.